Precision Medicine: New Frontiers for Rare Diseases

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Rare Disease Legislative Advocates in coordination with Rare Disease Congressional Caucus Co-Chairs:

Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY), will host a briefing on:
Precision Medicine: New Frontiers for Rare Diseases

Thursday, November 5th, 2015
12:00 pm – 1:00 pm (lunch provided)
Rayburn House Office Building
Room: B318 

speakers for precision Medicine briefing

Moderator: John Crowley, Chairman & CEO, Amicus Therapeutics

  • Matt Might, President, NGLY1.org & Associate Professor, University of Utah
  • Erynn Gordon, Director of Clinical Development, 23andMe
  • John Stone, Counsel, Committee on Energy and Commerce – U.S. House of Representatives
  • Dr. Christopher Austin, Director, National Center for Advancing Translational Sciences, NIH
  • Sean Sigmon, Business Development Director, Oracle Health Sciences

Limited Seating, RSVP to attend

Can’t attend? Click here to contact your congressional office to ask them to send a representative!

Hosted by:

Precision Medicine Logo Mashup

Rare Disease Legislative Advocates’ North-East Conference

 

Monday, July 20, 2015

Stanley Bergen Building, Rutgers University, Newark, NJ

Rare disease patient advocates and other stakeholders from the rare disease community came together on Monday, July 20th, 2015 in Newark, NJ for our inaugural North-East Legislative Conference. The goal of our new regional legislative conferences was to update advocates about important legislation advancing through Congress and to prepare them to meet with their Representatives during August Recess.
This year Congress is working on legislation that will have a huge impact on drug development and approval, and as a community we must join together to ensure the legislation benefits rare disease patients and is passed in both the House and the Senate.

 

 

This event was a great success view photos here:
RDLA-NEConf-talking rare diseases
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RDLA-NEConf-pallone in audienceRDLA-NEConf-lanceRDLA-NEConf-lance advocatesRDLA-NEConf-juliapalloneschillionsRDLA-NEConf-eatingRDLA-NEConf-dvocacy panelRDLA-NEConf-andy and advocatesDean P NE confNE Conf hinj
Pallone speaking Jill Wood Rutgers dean
Mike Illions thumbs up
Co-Hosted by: 

 

Thank you to our sponsors:NJ Leg Conf Logo Mashup 7.13.3

Rare Disease Congressional Caucus Briefing – 21st Century Cures Initiative: Priorities for the Rare Disease Community

View the Presentations and photos from 2015 Legislative Conference

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Click Here to view presentations and photos from this year’s Rare Disease Legislative Conference:

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Rare Disease Congressional Caucus Briefing: Urgent Healthcare Policy Needs of the Rare Disease Community

Rare Disease Legislative Advocates & the National MPS Society in coordination with Rare Disease Congressional Caucus Co-Chairs:
Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY), held a briefing,

Urgent Healthcare Policy Needs of the Rare Disease Community

Thursday, February 26th, 2015

Russell Caucus Room

Lunch Provided by event sponsors AbbVie, Alexion, Raptor, & Shire
Moderator:  Anthony J. Castaldo, President, US Hereditary Angiodema Association (HAEA)
  • Limited Access to Specialists in Private Insurance Networks, Stephanie Bozarth, National MPS Society
  • Coverage Gaps for Medically Necessary Foods, Nicole Dreyer-Gavin, PKU Parent Advocate
  • Medicare Coverage Challenges for Cystinosis Patients, Kristina Broadbelt, Patient Advocate
  • Step-wise Therapies: Negative Implications for the Health of Rare Disease Patients, Anthony J. Castaldo, President, US Heriditary Angiodema Association

 VIEW THE BRIEFING HERE

Briefing Hosted by:

 

October RDLA Meeting

Please join us for an
Advocates Luncheon/Conference Call

Thursday, October 16th, 2014
12 noon to 1 p.m. EST

Location:
RDLA DC Office
1101 14th Street NW, Suite 700
Washington, DC,  20005

Lunch will be provided

Click the titles to view PowerPoint Sides:

Please click here to listen to the audio recording of the call

Mark your Calendars!

BIO Patient & Health Advocacy Summit: Oct 14-15 2014, in DC

 

BIO is throwing their Patient & Health Advocacy Summit, titled “The Power of Partnerships” this October 14-15 in Washington, DC.

The event voluntary brings health organizations and the biotechnology industry together for two days of policy-driven panel discussions, best practice seminars and invaluable networking opportunities. The summit will feature speeches from Janet Woodcock, who serves as Director of the FDA center for Drug Evaluation, Representatives Diana Degette (D-CO) and Joe Pitts (R-PA), and many more.

To register and find out more, clickbit.ly/biosummit

Rare Diseases Congressional Caucus Briefing Sept.17th

Rare Disease Legislative Advocates & Kids v Cancer in coordination with Rare Disease Congressional Caucus Co-Chairs
Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY), will host a briefing

“Implementation of the Rare Disease Provisions in the
Food & Drug Administration Safety and Innovation Act of 2012 (FDASIA) ”
Read more

Nominate someone today for a RareVoice Award!

 

Rare Disease Legislative Advocates is pleased to open nominations for the 3rd Annual rarevoicelogoweb

A celebration to honor advocates who give rare disease patients a voice on Capitol Hill

Thursday, November 13, 2014 Arena Stage in Washington, DC

www.RareVoiceAwards.org RareVoice Award nominations are open to the public.  We encourage the community to nominate individuals and organizations who advocate for legislation and policy that benefits the rare disease community.  If you know of someone who has been a “Voice” for the rare disease community and should be honored for their work, please click here to submit your nomination.  We have nominations open for three award categories, Member of Congress or Congressional Staff, Patient Advocate or Patient Organization, and Government Agency Staff.  Deadline to submit nominations is  Friday August 29, 2014. Sponsorship Opportunities are available, contact Julia@RareAdvocates.org.   Thank you to our sponsors:

Gala Sponsor Mashup

Rare Disease Congressional Caucus Briefing

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Rare Disease Legislative Advocates in coordination with Rare Disease Congressional Caucus Co-Chairs

Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY), will host a briefing on

Implementation of the Rare Disease Provisions in the
Food & Drug Administration Safety and Innovation Act of 2012 (FDASIA) PL 112-144

Wednesday, September 17, 2014

12:00 noon – 1:30 pm

Rayburn House Office Building, Room: B-318

Lunch Provided by event sponsors Amicus & Shire

Agenda:

  • Sec. 901. Enhancement of accelerated patient access to new medical treatments: Accelerated Approval for rare diseases, Emil Kakkis, MD PhD, EveryLife Foundation for Rare Diseases
  • Sec. 902. Breakthrough therapies, TBD, Novartis Therapeutics
  • Sec. 903. Consultation with external experts on rare diseases, Stephanie Krenrich, Cystic Fibrosis Foundation
  • Sec. 908. Rare pediatric disease priority review voucher incentive program, Nancy Goodman, Kids v Cancer
  • Sec. 1137. Patient Participation in Medical Product Discussions, Allison Greenstein, Narcolepsy Patient Advocate
  • FDA’s Strategic Plan for Accelerating the Development of Therapies for Pediatric Rare Diseases – TBD, FDA
  • Emerging Biotech Perspective of the combined impact of FDASIA, TBD, Amicus Therapeutics

Limited Seating, RSVP to attend: To register click here.

Briefing Hosted by:

Sept. Caucus briefing logo mashup